B ENEFITS DEFINED BY TECHNOLOGY APPRAISALS

CHAPTER 10 UNITED KINGDOM

10.3 B ENEFITS DEFINED BY TECHNOLOGY APPRAISALS

Within the regulatory framework of laws and quasi-laws, there is large usage of HTA. In most cases, however, there is no statutory obligation for NHS bodies to implement standards. An exception is the status of technology appraisals. The NHS is legally obliged to fund and resource medicines and treatments recommended by NICE’s technology appraisals (National Institute for Health and Clinical Excellence, 2005b). The secretary of state for health has specified that if a technology appraisal states that a new technology should be made available, NHS bodies must ensure under the National Health Services Act 1977 that the technology is available not later than three months from the date of publication of that technology appraisal (Secretary of State for Health, 2001). Because of this formalized arrangement, this chapter describes in detail the evaluative approach that underlies technology appraisals.

10.3.1 Policy

Technology appraisals are undertaken by NICE with the purpose of considering the evidence of the health benefits and costs of a technology. The Institute’s Appraisal Committee considers the evidence and judges whether or not the technology can be recommended as a cost-effective use of NHS resources. The Appraisal Committee is a standing advisory committee. It seeks the views of many stakeholders, but its advice is independent. The Appraisal Committee’s judgment is published as the Appraisal Consultation Document. After comments are received from consultees and commentators, the appraisal sets out its final recommendations in the Final Appraisal Determination. The Appraisal Program Director (responsible for the whole Appraisal Program) and the Executive Lead (a member of the Appraisal Committee who is responsible for this particular appraisal) oversee the project, ensuring that the appraisal is conducted in accordance with the published guidelines for the appraisal process, signing of consultation documents in various stages of the appraisal process. They also undertake a final review of the Final Appraisal Determination, sign it off, and submit a report to the institute’s Guidance Executive, who will approve it for publication. Next, NICE will issue guidance to the NHS in England and Wales.

The Department of Health and the Welsh Assembly Government are responsible for selecting topics for the NICE technology appraisal and clinical guidelines work programs, using published criteria (National Institute for Health and Clinical Excellence, 2005b). Healthcare professionals, patients and carers and the general public who suggest topics read these criteria before submitting their suggestion. The types of technologies suggested include: pharmaceuticals, medical devices, diagnostic techniques, surgical procedures, other therapeutic technologies, and health promotion activities. The Advisory Committee on Topic Selection produces a potential list of topics. The Ministers for Health decide which technologies are referred to NICE review. In general, the presumption is that guidance will take the form of a clinical guideline if no suitable guidance relating to the condition is available or in preparation. An appraisal

should be considered if the perceived need relates to a particular intervention for a particular condition, and if either (a) there is an urgent need for guidance or (b) a clinical guideline for that condition is already available or in preparation (National Institute for Health and Clinical Excellence, 2005b).

As said above, when a technology appraisal recommends the use of a particular technology, NHS organizations in England and Wales are required to provide funding for this technology within 3 months.

Local NHS organizations are expected to meet the costs of those treatments out of their general annual budgets. The NHS agencies are accountable to the Department of Health. NICE tracks the progress of NHS trusts in implementing their guidance to ensure NICE guidance achieves positive and sustainable changes for patients, and to inform the development of future tools and resources. They have for example commissioned several surveys to track the uptake of NICE guidelines across the country.

To perform its various tasks, NICE has a staff of about 230 people in London and Manchester (personal communication with NICE). The staff grew exponentially over the last two years. Most of the Institute’s funding comes from the Department of Health. In 2005/6 NICE received £27m. It also received £0.6m from the Welsh Assembly Government and £0.1m from other sources. Main areas of expenses are staff salaries (accounting for about 42% of expenses in 2005) and external contracts (38%) (National Institute for Health and Clinical Excellence, 2006). The overall budget is split between work programs (the Center for Clinical Practice accounts for 27% of resource use, the Center for Health Technology Appraisals for 10%) and support functions (18% of the budget is used for planning and resources, 14% for communications, and 11% for implementation).

10.3.2 Assessment

The purpose of the appraisal is to appraise health benefits (quality of life, mortality) and costs of a technology and make recommendations to the NHS in England and Wales, as described in directions of the Secretary of State for health and the Welsh Assembly Government. The appraisal of a health technology is divided in three distinct phases: scoping, assessment, and appraisal (National Institute for Health and Clinical Excellence, 2004a). Information about the scoping, assessment and appraisal procedures is published on the Internet. During the scoping process, NICE determines what questions need to be addressed in the appraisal and developes a final scoping document and a protocol for the technology assessment. Components of the scope for example include definition of the patient population and relevant subgroups, choice of the comparator technologies, and methods of the economic evaluation (e.g., outcomes measure, cost categories, perspective). The scoping process is done for a long list of technologies. The Minister of Health decides which of those technologies will be referred for NICE Technology Appraisal. The assessment procedure that follows will be done within the boundaries defined in the scoping process. The appraisal procedure includes other factors relevant to the NICE recommendation, based on guidelines received from the policy makers (see below).

When a technology is referred to NICE, the Appraisal Committee undertakes the evaluation, starting a process in which scientific evidence and patient/professional expert opinion are systematically collected for the purpose of making NICE recommendations. The evaluation of scientific evidence, i.e.

technology assessment, is subcontracted to the assessment group, which is an independent academic body.

When the assessment report is received, the Appraisal Committee considers the outcomes of the assessment process, within the context of additional information supplied by consultees, commentators, clinical specialists and patient experts, who have also received a copy of the assessment report. The Appraisal Committee considers the evidence and formulates the NICE recommendations, i.e. whether or not the technology can be recommended for general use, or for use in specific groups. This judgement is referred to as Appraisal Determination. Since the Appraisal Committee is a key actor, its members are carefully selected. To ensure that evidence is evaluated independently and from a range of perspectives, members are appointed for three years, and are drawn from the NHS, patient and carer organizations, academia, and industry.

A consultation process has been designed to allow stakeholders to participate in the appraisal process as consultees or commentators. Consultees and commentators are identified relatively early in the technology appraisal, so that they can be involved in all stages of the appraisal. Consultees are:

manufacturers or sponsors of a technology, national professional organizations, national patient organizations, the Department of Health and the Welsh Assembly Government, relevant NHS organizations in England and local health boards in Wales. Manufacturers of comparator technologies participate in the process as commentators. They are not asked to offer their views and, contrary to consultees, they do not have the right to appeal to a final decision. Also other organizations may be invited to participate as commentators, like the NHS Quality Improvement Group in Scotland, National Collaborating Centers, and other related research groups. When a technology first appears at a provisional list of technologies that may be referred to NICE, already organizations are identified who might wish to participate in the appraisal process as consultees and commentators. They are invited to offer their views on the draft scope of the appraisal. When the technology is referred to NICE, they will also be invited to offer their views on the assessment report, and the appraisal consultation document. In addition, consultees can nominate specialist and patient experts to present their views to the appraisal committee.

They are also given the opportunity to appeal. Commentators are not asked to make a submission, and have no opportunity to appeal.

The evidence considered in the assessment and appraisal processes concerns health benefits (quality of life, mortality) and costs of a technology, as requested in the scoping documents. The assessment group performs a literature review and an economic evaluation. NICE offers methodological guidance, particularly where the economic evaluation is concerned, because of general awareness that methods of particularly economic evaluations are continuously evolving, and that there may be a shortage of the skills required to make assessments. A reference case is adopted that was considered appropriate for the Appraisal Committee’s purpose. The timelines for the assessment are not fixed, because different technologies require different assessments as decided upon in the scoping phase. For each technology, however, the timelines of the whole appraisal procedure will be presented on the website of NICE within 6 weeks of referral of that technology. Table 10-1 gives an approximation of the timelines.

Table 10-1

Weeks (approx.) since process began Step 1 Organizations invited to participate in the appraisal as consultees or

commentators

Step 2 Submissions from consultees received 14

Step 3 Submissions from manufacturers and sponsors sent to the Assessment Group 15 Step 4 Selected clinical specialists and patient experts invited to attend Appraisal

Committee meeting and asked to submit a written personal perspective

Step 5 Assessment Report received by Institute 28

Step 6 Assessment Report sent to consultees and commentators for comment 30 Step 7 Selected clinical specialists and patient experts submit written personal

perspectives

Step 8 Comments on Assessment Report from consultees and commentators received by Institute

Step 9 Evaluation Report compiled and sent to Appraisal Committee 36 Timelines may change in response to individual appraisal requirements.

The assessment group publishes its findings in an assessment report that is circulated. Comments are collected from consultees and commentators. After this the Appraisal Committee considers the evidence and comments and summarizes its findings in an Evaluation Report that will be used in the Appraisal process (described in the next section). The evaluation report contains:

• The final scope and lists of consultees and commentators;

• The assessment report;

• Comments of consultees and commentators on the assessment report;

• An overview by the Institute’s technical Lead for the Appraisal;

• Submissions from consultees;

• Summaries of submissions from commentators;

• Perspective of pateitns and clinical experts obtained in the Appraisal Committee meeting;

• The assessment Group’s written response to comments, if any, on the assessment report;

• Supplementary analysis, if any.

10.3.3 Decision

The appraisal stage of the process consists of four elements (National Institute for Health and Clinical Excellence, 2004b):

• consideration of the evidence at an Appraisal Committee meeting to develop an ACD;

• preparation of and consultation on the ACD;

• review of the ACD in the light of comments from consultation at a second Appraisal Committee meeting;

• preparation of the FAD.

The Appraisal Committee evaluates the evidence presented in the assessment report, taking the opinion of consultees and commentators into account. On basis of the assessment report and comments, the Appraisal Committee then develops an Evaluation Report that is circulated for the Appraisal Committee meeting in which the content of the Appraisal Consultation Document is discussed. At this moment, also factors beyond the health benefits and costs are taken into account, based on directions provided by the Secretary of State for health and the Welsh Assembly Government. These factors include clinical priorities, clinical need, balance of benefits and of costs, available resources, effective use thereof, and a long-term interest of the NHS in innovation. Next the Appraisal Consultation Document is prepared.

What follows is a consultation of the Appraisal Consultation Document, after which in a second meeting of the appraisal committee the content of the Final Appraisal Determination is discussed. Finally, the Final Appraisal Determination will be prepared. When the Final Appraisal Determination is signed off, it is circulated to NHS trusts, who are obliged to follow included recommendations. The timelines of the appraisal procedure are summarized in Table 10-2.

Table 10-2

Weeks (approx.) since process began Step 10 Appraisal Committee meeting to develop an ACD, attended by patient

experts and clinical specialists

Step 11 ACD produced and distributed. ACD posted on Institute’s website 5 working days later

Step 12 Fixed 4-week consultation period on ACD 40-43

Step 13 Appraisal Committee meeting to consider comments on ACD from consultees and commentators and comments received through the consultation on the Institute’s website. Appraisal Committee agrees the content of the FAD

Step 14 FAD produced and distributed. FAD posted on Institute’s website 5 working days later

51 Timelines may change in response to individual appraisal requirements.

The Appraisal process requires two meetings of the Appraisal Committee. The first meeting is attended by representatives of the assessment group and representatives from the National Collaborating Center that is responsible for developing the Institute’s clinical guidelines in areas related to the topic under appraisal.

Patient experts and clinical specialists are asked to submit, before the meeting, a written personal view of the role of the technology, particularly providing insights not available in the published literature. They are also invited to the meeting and get the opportunity to comment on the technology and its use in the NHS.

They are asked to withdraw from the committee before the contents of the Appraisal Consultation Document are discussed. Assessment Group representatives may remain present during Committee discussions in order to answer any further questions the Committee may have. However, they play no part

in decision-making. Representatives from the National Collaborating Center observe and serve as advisors to the Committee.

The Appraisal Consultation Document contains provisional recommendations that may change in response to consultation. The Appraisal Consultation Document is normally circulated three weeks after the first Appraisal Committee meeting. The document contains for example summaries of available evidence, and a description of clinical need and practice in the disease area. It is sent out to consultees and commentators, together with the Evaluation Report. Consultees and commentators have four weeks to submit their comments in writing. Purpose is to gather views on suggested recommendations and to determine whether recommendations are an appropriate interpretation of the evidence. New data will be collected only by prior agreement of the Appraisal Program Director, and only when it would significantly affect recommendations. When this happens the next meeting may be postponed. It is also possible that new data require substantial revision of interpretations and then a second Appraisal Consultation Document may be prepared, and the whole appraisal process is repeated. When views on the Appraisal Consultation Document have been collected, the second meeting of the Appraisal Committee is held to develop the Final Appraisal Determination. Representatives of the assessment group and the National Collaborating Center are invited to attend the meeting. To deal with issues raised during the consultation period, the chair may also invite experts. This meeting decides on the contents of the Final Appraisal Determination, which is normally published six weeks after the meeting when it is signed off by the Guidance Executive. It is also circulated to consultees and commentators, who in addition receive a summary of comments and a summary of the response to the comments. This information is also posted on the website.

10.3.4 Outputs and implementation

When the Final Appraisal Determination is finished, there is possibility for appeal. Right to appeal is limited to consultees. Appeals must be made within 3 weeks after receipt of the document. Appeals will only be considered when the grounds for appeal are appropriate, i.e. the institute has not acted in accordance with published procedures; the recommendations are ‘perverse’ in light of available evidence;

the institute has exceeded its powers. The appeal procedure, which has been set out in detail in a document (National Institute for Health and Clinical Excellence, 2004c), should not be regarded as an opportunity to reopen the argument. Normally, when an appeal is made, an Appeal Panel will be installed.

Members of the Appeal Panel include at least 5 people of the Appraisal Committee, but they will have had no previous involvement in the appraisal in question. The Appeal Panel considers the appeal in public.

This panel sends its conclusions to the institute. The Guidance Executive considers the panel’s decision and may decide to refer the Final Appraisal Determination back to the Appraisal Committee. If there is no appeal, an appeal is dismissed, or an appeal is upheld but the FAD does not need to be referred back to the Appraisal Committee (e.g., when only editorial changes are required), the Institute makes arrangements for the guidance to be published, together with the Appeal Panel decision, if any.

Publication takes place on the fourth Wednesday of each month. If the Final Appraisal Determination is

referred back to the Appraisal Committee, the Appeal Panel’s decision will also be published along with the arrangements for considerations by the Committee. This happens within 3 weeks of consideration by the Guidance Executive.

When NICE publishes its recommendations, it will also indicate when guidance or technology appraisals will be considered for reviews. The decision depends on the anticipated rate of developments in the field, and varies between 1 and 5 years. If significant evidence emergences sooner, consultees, commentators and the institute can identify it. The Guidance Executive considers the evidence and decides if the review date will be brought forward. A review will not be undertaken within 12 months of first publication of NICE recommendations.

To facilitate implementation and inform the public, NICE produces three versions of its technology appraisals (National Institute for Health and Clinical Excellence, 2005b):

• The full appraisal: presents the recommendations from the full version in a format suited to implementation by health professionals and NHS bodies;

• A quick reference guide: presents recommendations in a suitable format for health professionals;

• A lay version: information for the public is written for using suitable language for people without specialist medical knowledge.

Once NICE publishes its guidance, health professionals and organizations are expected to take the information fully into account in deciding what treatments to give to patients. NICE guidance generally acts as quasi-law: health professionals and organizations are expected to compare their clinical practice to the guidelines. Where NICE technology appraisals are concerned, the NHS has a statutory duty to provide funding and resources for the technology in question. To help ensure that NICE guidance is put into practice, NICE has established an Implementation Directorate that for example develops support tools, demonstrates cost impacts, evaluates guidance uptake, and ensures intelligent dissemination to the target audiences. NICE reports on its website the activities that are undertaken to evaluate whether or not its guidance is put into practice. A large effort was made in 2004, when NICE commissioned Abacus to measure the impact of 28 individual pieces of NICE guidance (Howard & Harrison, 2005). Another agency involved in testing implementation of NICE guidance is the Healthcare Commission. The Healthcare Commission’s statutory duty is to assess the performance of healthcare organizations in the NHS. Their activities include an annual health check, programs of review, audit and assessment. Amongst other things they assess how NHS organizations perform against a set of standards for NHS services. One

In document International comparison of systems to determine entitlements to medical specialist care: performance and organizational issues (Page 136-143)